[go: up one dir, main page]

WO2013096899A3 - Formulations stables pour l'administration au système nerveux central d'arylsulfatase a - Google Patents

Formulations stables pour l'administration au système nerveux central d'arylsulfatase a Download PDF

Info

Publication number
WO2013096899A3
WO2013096899A3 PCT/US2012/071473 US2012071473W WO2013096899A3 WO 2013096899 A3 WO2013096899 A3 WO 2013096899A3 US 2012071473 W US2012071473 W US 2012071473W WO 2013096899 A3 WO2013096899 A3 WO 2013096899A3
Authority
WO
WIPO (PCT)
Prior art keywords
arylsulfatase
cns delivery
stable formulations
asa protein
rhasa
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Ceased
Application number
PCT/US2012/071473
Other languages
English (en)
Other versions
WO2013096899A2 (fr
Inventor
Nazila Salamat-Miller
Katherine Taylor
Paul Campolieto
Zahra Shahrokh
Jing Pan
Lawrence Charnas
Teresa Leah Wright
Pericles Calias
Keethkumar JAIN
Sujit Basu
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Shire Human Genetics Therapies Inc
Original Assignee
Shire Human Genetics Therapies Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Shire Human Genetics Therapies Inc filed Critical Shire Human Genetics Therapies Inc
Priority to US14/368,153 priority Critical patent/US20140377244A1/en
Priority to EP12859411.6A priority patent/EP2793922B1/fr
Publication of WO2013096899A2 publication Critical patent/WO2013096899A2/fr
Publication of WO2013096899A3 publication Critical patent/WO2013096899A3/fr
Anticipated expiration legal-status Critical
Priority to US16/000,557 priority patent/US10660944B2/en
Priority to US16/851,822 priority patent/US20200237883A1/en
Ceased legal-status Critical Current

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/51—Lyases (4)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
    • A61K47/10—Alcohols; Phenols; Salts thereof, e.g. glycerol; Polyethylene glycols [PEG]; Poloxamers; PEG/POE alkyl ethers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/19—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles lyophilised, i.e. freeze-dried, solutions or dispersions
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/06—Sulfuric ester hydrolases (3.1.6)
    • C12Y301/06008—Cerebroside-sulfatase (3.1.6.8)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Dermatology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Psychology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Enzymes And Modification Thereof (AREA)

Abstract

La présente invention concerne, entre autres choses, des compositions et des procédés pour l'administration au système nerveux central d'enzymes lysosomales (par exemple, arylsulfatase A humaine recombinante (rhASA)) pour un traitement efficace de maladies de stockage lysosomal (par exemple, maladie de leucodystrophie métachromatique). Dans certains modes de réalisation, la présente invention comprend une formulation stable pour une administration intrathécale comprenant une protéine ASA et un poloxamère, où moins de 3 % de la protéine ASA existe sous forme agrégée.
PCT/US2012/071473 2011-12-23 2012-12-21 Formulations stables pour l'administration au système nerveux central d'arylsulfatase a Ceased WO2013096899A2 (fr)

Priority Applications (4)

Application Number Priority Date Filing Date Title
US14/368,153 US20140377244A1 (en) 2011-12-23 2012-12-21 Stable formulations for cns delivery of arylsulfatase a
EP12859411.6A EP2793922B1 (fr) 2011-12-23 2012-12-21 Formulations stables pour l'administration au système nerveux central d'arylsulfatase a
US16/000,557 US10660944B2 (en) 2011-12-23 2018-06-05 Stable formulations for CNS delivery of arylsulfatase A
US16/851,822 US20200237883A1 (en) 2011-12-23 2020-04-17 Stable formulations for cns delivery of arylsulfatase a

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201161580064P 2011-12-23 2011-12-23
US61/580,064 2011-12-23

Related Child Applications (2)

Application Number Title Priority Date Filing Date
US14/368,153 A-371-Of-International US20140377244A1 (en) 2011-12-23 2012-12-21 Stable formulations for cns delivery of arylsulfatase a
US16/000,557 Continuation US10660944B2 (en) 2011-12-23 2018-06-05 Stable formulations for CNS delivery of arylsulfatase A

Publications (2)

Publication Number Publication Date
WO2013096899A2 WO2013096899A2 (fr) 2013-06-27
WO2013096899A3 true WO2013096899A3 (fr) 2013-09-06

Family

ID=48669718

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2012/071473 Ceased WO2013096899A2 (fr) 2011-12-23 2012-12-21 Formulations stables pour l'administration au système nerveux central d'arylsulfatase a

Country Status (3)

Country Link
US (3) US20140377244A1 (fr)
EP (1) EP2793922B1 (fr)
WO (1) WO2013096899A2 (fr)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PT3626258T (pt) 2010-06-25 2021-10-19 Shire Human Genetic Therapies Métodos e composições para fornecimento ao snc de iduronato-2-sulfatase
TWI752907B (zh) 2015-05-08 2022-01-21 美商拜奧馬林製藥公司 用於治療cln2疾病之tpp1調配物及方法
IL261246B2 (en) 2016-02-24 2023-03-01 Biomarin Pharm Inc Therapeutic fusion proteins targeting lysosomal enzymes, their formulations and their uses
US12076332B2 (en) 2018-04-27 2024-09-03 The Regents Of The University Of California Treatment of lysosomal storage disorders
WO2020227166A1 (fr) * 2019-05-03 2020-11-12 The Trustees Of The University Of Pennsylvania Compositions utiles dans le traitement d'une leucodystrophie métachromatique
SI4021575T1 (sl) 2019-08-29 2024-10-30 Biomarin Pharmaceutical Inc. Metode za zdravljenje bolezni CLN2 pri prediatričnih subjektih
US20230090654A1 (en) * 2021-08-24 2023-03-23 Homology Medicines, Inc. Adeno-associated virus formulations
WO2024226471A2 (fr) 2023-04-24 2024-10-31 Biomarin Pharmaceutical Inc. Compositions et méthodes de traitement de troubles liés à stxbp1

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100221235A1 (en) * 2006-02-08 2010-09-02 Diatos Compositions and Methods for Treating Lysosomal Storage Diseases
WO2011041897A1 (fr) * 2009-10-06 2011-04-14 Angiochem Inc. Compositions et procédés pour transporter des agents thérapeutiques
US20110300120A1 (en) * 2008-12-16 2011-12-08 Avila Luis Z Oligosaccharide-protein conjugates

Family Cites Families (48)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4743265A (en) 1986-04-23 1988-05-10 Dij Catheter Corp Articulated catheter placement device
US5863782A (en) 1995-04-19 1999-01-26 Women's And Children's Hospital Synthetic mammalian sulphamidase and genetic sequences encoding same
US6118045A (en) 1995-08-02 2000-09-12 Pharming B.V. Lysosomal proteins produced in the milk of transgenic animals
ATE225184T1 (de) 1997-08-22 2002-10-15 Seikagaku Kogyo Co Ltd Arzneimittel zur behandlung von durch hernie gestörter intervertebraler bandscheibe
WO2000034451A1 (fr) 1998-12-07 2000-06-15 Pharming Intellectual Property B.V. Traitement de la maladie de pompe
US6217552B1 (en) 1999-03-01 2001-04-17 Coaxia, Inc. Medical device for selective intrathecal spinal cooling in aortic surgery and spinal trauma
US20020052311A1 (en) 1999-09-03 2002-05-02 Beka Solomon Methods and compostions for the treatment and/or diagnosis of neurological diseases and disorders
US6534300B1 (en) 1999-09-14 2003-03-18 Genzyme Glycobiology Research Institute, Inc. Methods for producing highly phosphorylated lysosomal hydrolases
US20020099025A1 (en) 1999-12-30 2002-07-25 Heywood James A. Treatment of neurological disorders
US6866844B2 (en) 2002-11-07 2005-03-15 Biomarin Pharmaceutical Inc. Precursor N-acetylgalactosamine-4-sulfatase, methods of treatment using said enzyme and methods for producing and purifying said enzyme
US7560424B2 (en) 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
US20040005309A1 (en) 2002-05-29 2004-01-08 Symbiontics, Inc. Targeted therapeutic proteins
ATE384736T1 (de) 2001-04-30 2008-02-15 Zystor Therapeutics Inc Subzelluläres targeting von therapeutischen proteinen
US7629309B2 (en) 2002-05-29 2009-12-08 Zystor Therapeutics, Inc. Targeted therapeutic proteins
US20030072761A1 (en) 2001-10-16 2003-04-17 Lebowitz Jonathan Methods and compositions for targeting proteins across the blood brain barrier
WO2003032913A2 (fr) 2001-10-16 2003-04-24 Symbiontics Inc. Procedes et compositions pour le ciblage de proteines a travers la barriere hemato-encephalique (bhe)
JP5570677B2 (ja) 2002-04-25 2014-08-13 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド α−ガラクトシダーゼＡ欠損症の治療
EP1514106A4 (fr) 2002-05-29 2007-05-09 Zystor Therapeutics Inc Proteines therapeutiques ciblees
US20040248262A1 (en) 2003-01-22 2004-12-09 Koeberl Dwight D. Constructs for expressing lysomal polypeptides
US20050026823A1 (en) 2003-06-20 2005-02-03 Biomarin Pharmaceutical Inc. Use of the chaperone receptor-associated protein (RAP) for the delivery of therapeutic compounds to the brain and other tissues
CA2525236C (fr) 2003-06-20 2015-03-24 Biomarin Pharmaceutical Inc. Administration de composes therapeutiques au cerveau et a d'autres tissus
US7442372B2 (en) 2003-08-29 2008-10-28 Biomarin Pharmaceutical Inc. Delivery of therapeutic compounds to the brain and other tissues
NZ548871A (en) * 2004-01-30 2009-06-26 Shire Pharmaceuticals Ireland Use of arylsulfatase A for treating metachromatic leukodystrophy
WO2005074888A2 (fr) * 2004-02-03 2005-08-18 Biodelivery Sciences International, Inc. Structures cochleaires d'enzymes de remplacement
WO2005077093A2 (fr) 2004-02-06 2005-08-25 Biomarin Pharmaceutical Inc. Fabrication d'enzymes lysosomales hautement phosphorylees et utilisations associees
ES2344302T3 (es) 2004-02-10 2010-08-24 Zystor Therapeutics , Inc. Alfa glucosidasa acida y fragmentos de la misma.
US20050208090A1 (en) 2004-03-18 2005-09-22 Medtronic, Inc. Methods and systems for treatment of neurological diseases of the central nervous system
JPWO2006121199A1 (ja) 2005-05-11 2008-12-18 日本ケミカルリサーチ株式会社 脂質小胞体組成物
CN104771402A (zh) 2005-06-08 2015-07-15 阿米库斯治疗学公司 溶酶体酶编码基因突变相关的cns紊乱的治疗
AR059089A1 (es) 2006-01-20 2008-03-12 Genzyme Corp Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal
PL2631242T3 (pl) 2006-04-04 2023-03-20 Takeda Pharmaceutical Company Limited Sposób zatężania polipeptydu
GB0611463D0 (en) 2006-06-09 2006-07-19 Novartis Ag Organic compounds
US20080140056A1 (en) 2006-12-06 2008-06-12 Medtronic, Inc. Method for delivering large molecules to the brain
US20090041741A1 (en) 2007-03-06 2009-02-12 Saint Louis University Modified enzyme and treatment method
BRPI0815416A2 (pt) 2007-08-15 2014-10-21 Amunix Inc Composições e métodos para modificar propriedades de polipeptídeos biologicamente ativos
TWI661833B (zh) 2007-11-30 2019-06-11 百慕達商艾伯維生物技術有限責任公司 蛋白質調配物及製造其之方法
US7722865B2 (en) 2008-01-18 2010-05-25 Biomarin Pharmaceutical Inc. Manufacture of active highly phosphorylated human lysosomal sulfatase enzymes and uses thereof
US20110223147A1 (en) 2008-05-07 2011-09-15 Zystor Therapeutics, Inc. Lysosomal targeting peptides and uses thereof
EP3679942A1 (fr) 2009-06-17 2020-07-15 BioMarin Pharmaceutical Inc. Formulations pour des enzymes lysosomales
US8436489B2 (en) 2009-06-29 2013-05-07 Lightsail Energy, Inc. Compressed air energy storage system utilizing two-phase flow to facilitate heat exchange
MX2013000320A (es) 2010-06-25 2013-06-05 Shire Human Genetic Therapies Composiciones y metodos para suministro al sistema nervioso central de heparan n-sulfatasa.
DK2588130T3 (en) 2010-06-25 2016-10-24 Shire Human Genetic Therapies Cns delivery of therapeutic agents
NZ605873A (en) 2010-06-25 2015-02-27 Shire Human Genetic Therapies Methods and compositions for cns delivery of arylsulfatase a
HRP20191924T1 (hr) 2010-06-25 2020-01-10 Shire Human Genetic Therapies, Inc. Postupci i sastavi za isporuku iduronat-2-sulfataze u cns
PT3626258T (pt) 2010-06-25 2021-10-19 Shire Human Genetic Therapies Métodos e composições para fornecimento ao snc de iduronato-2-sulfatase
US20110318327A1 (en) 2010-06-25 2011-12-29 Shire Human Genetic Therapies, Inc. Treatment of sanfilippo syndrome type b
EP2588132A4 (fr) 2010-06-25 2014-10-15 Shire Human Genetic Therapies Procédés et compositions pour l'administration au snc de ss-galactocérébrosidase
JP2012062312A (ja) 2010-08-19 2012-03-29 Yoshikatsu Eto ハンター症候群の治療剤

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100221235A1 (en) * 2006-02-08 2010-09-02 Diatos Compositions and Methods for Treating Lysosomal Storage Diseases
US20110300120A1 (en) * 2008-12-16 2011-12-08 Avila Luis Z Oligosaccharide-protein conjugates
WO2011041897A1 (fr) * 2009-10-06 2011-04-14 Angiochem Inc. Compositions et procédés pour transporter des agents thérapeutiques

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
STROOBANTS, S ET AL.: "Intracerebroventricular Enzyme Infusion Corrects Central Nervous System Pathology And Dysfunction In A Mouse Model Of Metachromatic Leukodystrophy.", HUMAN MOLECULAR GENETICS., vol. 20, no. 14, 22 April 2011 (2011-04-22), pages 2760 - 2769, XP055157117 *

Also Published As

Publication number Publication date
EP2793922A4 (fr) 2015-10-28
EP2793922A2 (fr) 2014-10-29
US10660944B2 (en) 2020-05-26
US20200237883A1 (en) 2020-07-30
EP2793922B1 (fr) 2019-10-30
WO2013096899A2 (fr) 2013-06-27
US20190125841A1 (en) 2019-05-02
US20140377244A1 (en) 2014-12-25

Similar Documents

Publication Publication Date Title
WO2013096899A3 (fr) Formulations stables pour l'administration au système nerveux central d'arylsulfatase a
MX344795B (es) Composiciones y metodos para suministro al sistema nervioso central de arilsulfatasa a.
WO2011163649A3 (fr) Procédés et compositions pour la délivrance au snc d'iduronate-2-sulfatase
WO2011163647A3 (fr) Procédés et compositions pour une administration au snc d'héparane n-sulfatase
WO2011163651A3 (fr) Procédés et compositions pour l'administration au snc de ss-galactocérébrosidase
BR112012033214A2 (pt) método e composições para distribuição no snc de iduronato-2-sulfatase
AU2016204371A1 (en) Lyophilised and aqueous anti-CD40 antibody formulations
WO2010032140A3 (fr) Préparations pharmaceutiques et méthodes associées d'administration
MX2012002861A (es) Formulaciones farmaceuticas muy concentradas.
WO2010123569A3 (fr) Composés immunonanothérapeutiques fournissant une réponse à médiation par th1
NZ702801A (en) Treatment of sanfilippo syndrome type b
WO2012076670A3 (fr) Formulation d'anticorps
PH12014502778B1 (en) Antibody formulation
WO2013090278A3 (fr) Compositions intranasales de dexmédétomidine et leurs procédés d'utilisation
IN2015DN04151A (fr)
WO2011143271A3 (fr) Liposomes thérapeutiques et leurs procédés de production et d'utilisation
WO2012030308A3 (fr) Formulation comprenant la cellobiose
AR122036A2 (es) Composiciones farmacéuticas estables para la administración intratecal de arisulfatasa a
NZ608164A (en) Formulations for bovine granulocyte colony stimulating factor and variants thereof
WO2012015211A3 (fr) Composition de gouttes ophtalmiques contenant de la cyclosporine a
WO2012053758A3 (fr) Composition pharmaceutique destinée au traitement ou à la prévention de maladies du foie provoquées par l'alcool, contenant du cilostazol en tant que principe actif

Legal Events

Date Code Title Description
121 Ep: the epo has been informed by wipo that ep was designated in this application

Ref document number: 12859411

Country of ref document: EP

Kind code of ref document: A2

REEP Request for entry into the european phase

Ref document number: 2012859411

Country of ref document: EP

WWE Wipo information: entry into national phase

Ref document number: 2012859411

Country of ref document: EP

121 Ep: the epo has been informed by wipo that ep was designated in this application

Ref document number: 12859411

Country of ref document: EP

Kind code of ref document: A2